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A randomized, double blind, double-dummy placebo controlled, 3-way cross-over study to determine the test-retest reliability of, and the effect of oral retigabine and riluzole on, peripheral motor nerve excitability measurements in patients with ALS.

Patients with ALS are routinely prescribed riluzole, the only registered drug for the treatment of ALS. This study will evaluate the concentration-effect relationship of riluzole (100 mg) and retigabine (300 mg) on axonal excitability measures. In…

Ephedrine as add-on therapy for patients with myasthenia gravis

The main objective of this pilot study of n-of-one trials is to determine the effect of add-on treatment with ephedrine for all participants enrolled in this small series of n-of-one trials. Secondary objectives include determining the effect in…

Effects of ODM109 on respiratory function in patients with ALS. A
randomised, double blind, placebocontrolled, crossover, 3 period,
multicentre study with openlabel followup extension.

The primary objective of this study will investigate the effect of oral levosimendan on the respiratory function of patients with Amyotrophic Lateral Sclerosis.

A Multicenter, Randomized, Placebo-Controlled, Double-Blind Phase 1b Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of SAR443060 in Subjects with Amyotrophic Lateral Sclerosis (ALS)

Double-blinded part:The primary objectives of the study are as follows:• To investigate the safety and tolerability of 28 days of oral doses of SAR443060 in subjects with ALS.The secondary objectives of the study are as follows: • To characterize…

A Phase 2, randomised, double-blind, placebo-controlled, 2-way crossover study to evaluate the efficacy, safety, and tolerability of NMD670 in ambulatory adults with Type 3 spinal muscular atrophy

This study has been transitioned to CTIS with ID 2024-516321-31-00 check the CTIS register for the current data. The purpose of this study is to measure clinical efficacy on muscle strength and function, safety, and tolerability of NMD670 compared…

Improving symptomatic treatment with pyridostigmine and amifampridine: a randomized double-blinded, placebo controlled crossover trial in patients with myasthenia gravis (IMPACT-MG)

The aim of this study is to investigate the efficacy and tolerability of pyridostigmine monotherapy and amifampridine as add-on compared to placebo in patients with MG.

A randomised, double-blind, placebo controlled, single and multiple dose escalation study to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of NMD670 in male and female healthy subjects and patients with myasthenia gravis.

* To assess safety and tolerability of single oral doses of NMD670 in healthy male and female subjects * To assess safety and tolerability of repeated oral doses of NMD670 in healthy male subjects * To assess safety and tolerability of single oral…